Cell stem cell · 2023
Preclinical quality, safety, and efficacy of a human embryonic stem cell-derived product for the treatment of Parkinson's disease, STEM-PD.
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Kirkeby Agnete, Nelander Jenny, Hoban Deirdre B, Rogelius Nina, Bjartmarz Hjálmar, Novo Nordisk Cell Therapy R&D, Storm Petter, Fiorenzano Alessandro, Adler Andrew F, Vale Shelby, Mudannayake Janitha, Zhang Yu, Cardoso Tiago, Mattsson Bengt, Landau Anne M, Glud Andreas N, Sørensen Jens C, Lillethorup Thea P, Lowdell Mark, Carvalho Carla, Bain Owen, van Vliet Trinette, Lindvall Olle, Björklund Anders, Harry Bronwen, Cutting Emma, Widner Håkan, Paul Gesine, Barker Roger A, Parmar Malin
Аннотация
Cell replacement therapies for Parkinson's disease (PD) based on transplantation of pluripotent stem cell-derived dopaminergic neurons are now entering clinical trials. Here, we present quality, safety, and efficacy data supporting the first-in-human STEM-PD phase I/IIa clinical trial along with the trial design. The STEM-PD product was manufactured under GMP and quality tested in vitro and in vivo to meet regulatory requirements. Importantly, no adverse effects were observed upon testing of the product in a 39-week rat GLP safety study for toxicity, tumorigenicity, and biodistribution, and a non-GLP efficacy study confirmed that the transplanted cells mediated full functional recovery in a pre-clinical rat model of PD. We further observed highly comparable efficacy results between two different GMP batches, verifying that the product can be serially manufactured. A fully in vivo-tested batch of STEM-PD is now being used in a clinical trial of 8 patients with moderate PD, initiated in 2022.